Frontera Therapeutics has dosed the first participant in a Phase I clinical trial of FT-001 to treat Leber Congenital Amaurosis-2 (LCA-2), a severe inherited retinal disease that is related to a mutation in the RPE65 gene.
The Phase I clinical trial has been designed for assessing FT-001’s pharmacokinetics, therapeutic effects, tolerability, and safety as a single dose in LCA-2 patients.
It is being conducted in China and the initial data is anticipated in the second half of this year.
In April, the company obtained IND clearance for FT-001 from the US Food and Drug Administration (FDA).
Frontera Therapeutics CEO and founder Yong Dai said: “This is an exciting achievement for Frontera Therapeutics as we begin our first clinical trial and take a significant step forward as a company.”
The company’s lead AAV gene therapy product candidate, FT-001, is given through a one-time injection into the subretinal space of the eye.
It provides a functional copy of the human RPE65 gene to the nuclei of the retinal cells of the patient.
The addition of a functional copy of the RPE65 gene enables the expression of normal functioning human hRPE65 proteins, which improve the functional vision of a patient.
According to the company, LCA is classified as an ultra-orphan disease and is said to be found in two to three out of every 100,000 new borns.
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